MUSCULAR DYSTROPHIES

This study tests an oral drug called SAT-3247 in 51 boys aged 7 to 10 with Duchenne muscular dystrophy (DMD). The goal is to see if it can improve muscle strength and function over 12 weeks. Participants will take the drug or a placebo on weekdays, and all will continue their usu…

This study tests a brain implant that records and stimulates brain areas to help adults with paralysis control assistive devices like computers or wheelchairs and receive sensory feedback. The trial enrolls 5 people with conditions like spinal cord injury or ALS. The main goal is…

This program provides access to an investigational drug, AOC 1044, for people with Duchenne muscular dystrophy (DMD) whose genetic mutation can be treated by skipping exon 44. It is for eligible patients aged 6 and older living in the US. The goal is to offer treatment while gath…

This study is testing a device called mScan that uses a tiny, painless electrical current to measure muscle health. It takes about 6 seconds and is held against the skin. Researchers want to see if it can give similar results to an MRI, which is slower and more expensive. The goa…

This early-stage study tests a brain-computer interface (BCI) in up to 2 people who have lost the ability to speak due to conditions like spinal cord injury or ALS. The device is implanted in the brain to record signals when the person tries to speak, aiming to translate those si…

This early study tests whether a brain implant can help people with severe paralysis from conditions like ALS, spinal cord injury, or stroke control devices using their thoughts. The implant records brain signals to decode intended movements or speech, aiming to restore communica…

This early-stage study tests whether a brain implant can safely help people with severe paralysis (from ALS, spinal cord injury, or similar conditions) control a computer cursor or speech device just by thinking. Only 3 participants will be enrolled, and the main goal is to check…

This study aims to understand fatigue and walking patterns in people with neuromuscular diseases like muscular dystrophy, spinal muscular atrophy, and Charcot-Marie-Tooth disease. Researchers will measure walking speed during a 6-minute test and track daily activity with a wearab…